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2023 7
2024 2

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Page 1
PAM-flexible genome editing with an engineered chimeric Cas9.
Zhao L, Koseki SRT, Silverstein RA, Amrani N, Peng C, Kramme C, Savic N, Pacesa M, Rodríguez TC, Stan T, Tysinger E, Hong L, Yudistyra V, Ponnapati MR, Jacobson JM, Church GM, Jakimo N, Truant R, Jinek M, Kleinstiver BP, Sontheimer EJ, Chatterjee P. Zhao L, et al. Nat Commun. 2023 Oct 4;14(1):6175. doi: 10.1038/s41467-023-41829-y. Nat Commun. 2023. PMID: 37794046 Free PMC article.
PMS1 as a target for splice modulation to prevent somatic CAG repeat expansion in Huntington's disease.
McLean ZL, Gao D, Correia K, Roy JCL, Shibata S, Farnum IN, Valdepenas-Mellor Z, Rapuru M, Morini E, Ruliera J, Gillis T, Lucente D, Kleinstiver BP, Lee JM, MacDonald ME, Wheeler VC, Pinto RM, Gusella JF. McLean ZL, et al. bioRxiv [Preprint]. 2023 Jul 27:2023.07.25.550489. doi: 10.1101/2023.07.25.550489. bioRxiv. 2023. Update in: Nat Commun. 2024 Apr 12;15(1):3182. doi: 10.1038/s41467-024-47485-0. PMID: 37547003 Free PMC article. Updated. Preprint.
Base editing as a genetic treatment for spinal muscular atrophy.
Alves CRR, Ha LL, Yaworski R, Lazzarotto CR, Christie KA, Reilly A, Beauvais A, Doll RM, de la Cruz D, Maguire CA, Swoboda KJ, Tsai SQ, Kothary R, Kleinstiver BP. Alves CRR, et al. bioRxiv [Preprint]. 2023 Jan 21:2023.01.20.524978. doi: 10.1101/2023.01.20.524978. bioRxiv. 2023. PMID: 36711797 Free PMC article. Preprint.
Splice modulators target PMS1 to reduce somatic expansion of the Huntington's disease-associated CAG repeat.
McLean ZL, Gao D, Correia K, Roy JCL, Shibata S, Farnum IN, Valdepenas-Mellor Z, Kovalenko M, Rapuru M, Morini E, Ruliera J, Gillis T, Lucente D, Kleinstiver BP, Lee JM, MacDonald ME, Wheeler VC, Mouro Pinto R, Gusella JF. McLean ZL, et al. Nat Commun. 2024 Apr 12;15(1):3182. doi: 10.1038/s41467-024-47485-0. Nat Commun. 2024. PMID: 38609352 Free PMC article.
PAM-Flexible Genome Editing with an Engineered Chimeric Cas9.
Koseki S, Hong L, Yudistyra V, Stan T, Tysinger E, Silverstein R, Kramme C, Amrani N, Savic N, Pacesa M, Rodriguez T, Ponnapati M, Jacobson J, Church G, Truant R, Jinek M, Kleinstiver B, Sontheimer E, Chatterjee P. Koseki S, et al. Res Sq [Preprint]. 2023 Mar 7:rs.3.rs-2625838. doi: 10.21203/rs.3.rs-2625838/v1. Res Sq. 2023. Update in: Nat Commun. 2023 Oct 4;14(1):6175. doi: 10.1038/s41467-023-41829-y. PMID: 36945419 Free PMC article. Updated. Preprint.